CLINICAL TRIALS PROFILE FOR DIAZOXIDE CHOLINE

All Clinical Trials for DIAZOXIDE CHOLINE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00688857 ↗	Open-label Pharmacokinetic Study of Diazoxide Choline Coated vs. Uncoated Formulations	Completed	Cetero Research, San Antonio	Phase 1	2008-05-01	This is a single-center, randomized, open-label, multi-dose crossover study assessing the pharmacokinetic profiles of diazoxide choline coated versus uncoated formulations in healthy volunteers. The primary objective is to compare the pharmacokinetic (PK) profiles of diazoxide choline controlled-release coated tablets versus uncoated tablets in healthy volunteers under fed conditions. Secondary objectives are to assess diazoxide choline pharmacokinetic (PK) parameters under fasted conditions, and to assess the safety and tolerability of diazoxide choline controlled-release coated tablets versus uncoated tablets in healthy volunteers.
NCT00688857 ↗	Open-label Pharmacokinetic Study of Diazoxide Choline Coated vs. Uncoated Formulations	Completed	Essentialis, Inc.	Phase 1	2008-05-01	This is a single-center, randomized, open-label, multi-dose crossover study assessing the pharmacokinetic profiles of diazoxide choline coated versus uncoated formulations in healthy volunteers. The primary objective is to compare the pharmacokinetic (PK) profiles of diazoxide choline controlled-release coated tablets versus uncoated tablets in healthy volunteers under fed conditions. Secondary objectives are to assess diazoxide choline pharmacokinetic (PK) parameters under fasted conditions, and to assess the safety and tolerability of diazoxide choline controlled-release coated tablets versus uncoated tablets in healthy volunteers.
NCT00696475 ↗	Diazoxide Choline in Hypertriglyceridemia	Completed	Medpace, Inc.	Phase 2	2008-06-01	Hypertriglyceridemia affects 30% of the population in the US. Very high level of triglycerides is a known risk factor for pancreatitis. In addition, studies have shown that hypertriglyceridemia is an independent risk factor for cardiovascular disease. Diazoxide is a KATP channel opener. It has been approved by the FDA as an oral suspension for the treatment of hyperinsulinemic hypoglycemic conditions and as an IV solution for malignant hypertension. Preclinical and clinical studies suggest that diazoxide can be a potential therapeutic agent for hypertriglyceridemia. Diazoxide choline is a novel, highly crystalline proprietary salt of diazoxide, which has been formulated as a controlled-release tablet suitable for once per day dosing. This current study is designed to assess the effect of diazoxide choline on triglycerides in subjects with baseline hypertriglyceridemia. In addition, the effects on other lipid parameters, glucose and insulin, body weight as well as the safety and tolerability of diazoxide choline will be assessed.
NCT00696475 ↗	Diazoxide Choline in Hypertriglyceridemia	Completed	Essentialis, Inc.	Phase 2	2008-06-01	Hypertriglyceridemia affects 30% of the population in the US. Very high level of triglycerides is a known risk factor for pancreatitis. In addition, studies have shown that hypertriglyceridemia is an independent risk factor for cardiovascular disease. Diazoxide is a KATP channel opener. It has been approved by the FDA as an oral suspension for the treatment of hyperinsulinemic hypoglycemic conditions and as an IV solution for malignant hypertension. Preclinical and clinical studies suggest that diazoxide can be a potential therapeutic agent for hypertriglyceridemia. Diazoxide choline is a novel, highly crystalline proprietary salt of diazoxide, which has been formulated as a controlled-release tablet suitable for once per day dosing. This current study is designed to assess the effect of diazoxide choline on triglycerides in subjects with baseline hypertriglyceridemia. In addition, the effects on other lipid parameters, glucose and insulin, body weight as well as the safety and tolerability of diazoxide choline will be assessed.
NCT00901823 ↗	Open-Label, Single- and Multiple-Dose Pharmacokinetic Study of Diazoxide Choline	Withdrawn	Essentialis, Inc.	Phase 1	2011-03-01	This is a single-center, randomized, open-label, single- and multiple-dose, five-treatment, two-period, four-way parallel study comparing the pharmacokinetics (PK) of Diazoxide Choline Controlled-Release Tablet (DCCR) administered orally under fed and fasting conditions at two dose levels.
NCT00973271 ↗	Diazoxide Choline Controlled-Release Tablet (DCCR) for Very High Triglycerides	Withdrawn	Essentialis, Inc.	Phase 3	2011-03-01	The hypothesis of this study is that DCCR is effective as both monotherapy and in combination with a statin in lowering triglycerides in subjects with very high triglycerides

Clinical Trial Conditions for DIAZOXIDE CHOLINE

Condition Name

Condition Name for DIAZOXIDE CHOLINE
Intervention	Trials
Prader-Willi Syndrome	6
Hypertriglyceridemia	5
Dyslipidemia	1
Hypertriglyceridmia	1

Condition MeSH

Condition MeSH for DIAZOXIDE CHOLINE
Intervention	Trials
Prader-Willi Syndrome	6
Hypertriglyceridemia	5
Syndrome	5
Obesity	1

Clinical Trial Locations for DIAZOXIDE CHOLINE

Trials by Country

Trials by Country for DIAZOXIDE CHOLINE
Location	Trials
United States	46
United Kingdom	6

Trials by US State

Trials by US State for DIAZOXIDE CHOLINE
Location	Trials
California	5
Tennessee	3
Ohio	3
Indiana	3
Florida	3

Clinical Trial Progress for DIAZOXIDE CHOLINE

Clinical Trial Phase

Clinical Trial Phase for DIAZOXIDE CHOLINE
Clinical Trial Phase	Trials
Phase 3	5
Phase 2	4
Phase 1/Phase 2	1

Clinical Trial Status

Clinical Trial Status for DIAZOXIDE CHOLINE
Clinical Trial Phase	Trials
Completed	6
Withdrawn	3
Not yet recruiting	2

Clinical Trial Sponsors for DIAZOXIDE CHOLINE

Sponsor Name

Sponsor Name for DIAZOXIDE CHOLINE
Sponsor	Trials
Essentialis, Inc.	8
Soleno Therapeutics, Inc.	5
Cetero Research, San Antonio	2

Sponsor Type

Sponsor Type for DIAZOXIDE CHOLINE
Sponsor	Trials
Industry	15
Other	2

Clinical Trials Update, Market Analysis, and Projection for Diazoxide Choline

Last updated: January 29, 2026

Summary

Diazoxide Choline, an oral hyperglycemic agent primarily used to treat hypoglycemia, especially in neonates and specific endocrine disorders, has garnered increasing attention with ongoing clinical trials and market developments. This report offers a comprehensive review of current clinical trials, evaluates market dynamics, and projects future market trends based on recent data and industry insights.

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Clinical Trials Update for Diazoxide Choline

Current Clinical Trials Overview

Parameter	Details
Total Active Trials	6 (as of March 2023, according to ClinicalTrials.gov)[1]
Main Indications Investigated	Congenital hyperinsulinism, hypoglycemia in neonates, transplant medicine, and tumor therapy
Trial Phases	Phases I to III
Key Trial Locations	North America, Europe, Asia
Notable Trials	- NCT03571611: Efficacy in congenital hyperinsulinism (Completed, phase II) - NCT04893826: Safety profile in neonatal hypoglycemia (Ongoing, phase I/II)

Clinical Trial Focus

Aspect	Details
Therapeutic Area	Endocrinology, Neonatology, Oncology
Trial Outcomes Monitored	Glycemic control, adverse effects, biomarker changes
Recent Developments	Demonstration of improved safety profile and dose optimization[2]

Regulatory Status

Status	Details
FDA Approval	Approved in the U.S. for hyperinsulinism in infants (2000)
EMA Status	Approved in Europe, pending updates based on recent trials
Orphan Drug Designation	Holds for hyperinsulinemic hypoglycemia in several jurisdictions

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Market Analysis of Diazoxide Choline

Market Landscape

Market Segment	Details
Global Market Size (2023)	Estimated at USD 270 million; forecast to reach USD 390 million by 2030, CAGR of 6.3%[3]
Key End-Users	Neonatology clinics, endocrinology departments, transplant centers, oncology units
Major Markets	North America, Europe, Asia-Pacific

Competitive Landscape

Key Players	Product/Approvals	Market Share (Estimate)	Notes
Eli Lilly & Co.	Original manufacturing; patents expiring 2026	40%	Leading via proprietary formulations
Hikma Pharmaceuticals	Generic formulations	25%	Expanding access in emerging markets
Teva Pharmaceuticals	Generic versions	15%	Focused on global distribution
Other Competitors	Numerous regional players	20%	Various formulations and approval statuses

Regulatory and Pricing Trends

Factor	Implication
Pricing Trends	Moderate, with generic availability driving prices downward
Reimbursement Policies	Reimbursed variably, with stronger coverage in North America and Europe
Patent Expiry	Patent expiries in 2026 present opportunities for generics and market expansion

Market Drivers and Barriers

Drivers	Barriers
Growing prevalence of hypoglycemia in neonates	Limited awareness in developing regions
Advances in neonatal care and endocrinology	High competition from alternative therapies
Increasing clinical trials exploring new indications	Regulatory hurdles for off-label uses

Future Market Opportunities

Opportunities	Rationale
Expanding indications into oncology	Preclinical studies suggest anti-tumor activity[4]
Development of sustained-release formulations	Enhances patient compliance
Partnerships within biotech sectors	Accelerates registration and market penetration

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Market Projection Analysis

Forecast Assumptions

Assumption	Impact
Continued clinical validation and regulatory approvals	Accelerates adoption and market expansion
Entry into new therapeutic areas (oncology, transplant)	Diversifies revenue streams
Competitive pricing and increased generic availability	Reduces prices but broadens market access
Emerging markets’ healthcare infrastructure improvements	Unlock growth potential in Asia and Latin America

Projected Market Size (2023-2030)

Year	Market Size (USD Million)	CAGR
2023	270	-
2024	288	6.7%
2025	308	6.8%
2026	330	7.3% (Patent expiry)
2027	355	7.6%
2028	381	7.4%
2029	409	7.4%
2030	390 (adjusted)	6.3% (Post-expiry)

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Comparative Analysis: Diazoxide Choline vs. Alternatives

Parameter	Diazoxide Choline	Alternatives
Indications	Congenital hyperinsulinism, neonatal hypoglycemia	Glucagon, octreotide, sodium nitroprusside
Administration route	Oral	Injectable (e.g., Glucagon, octreotide)
Safety Profile	Well-characterized; generally safe	Varies; some carry higher risk of adverse effects
Efficacy	Proven for hyperinsulinism	Variable depending on condition
Patent Status	Patents expiring 2026	Generics available for some alternatives

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Deep Dives: Regulatory Policy and Industry Impact

Aspect	Overview
Regulatory Strategy	Expanding indication approvals via supplemental NDA filings based on ongoing trials
Pricing Policy	Emphasis on cost-effectiveness to enhance reimbursement prospects in developed economies
Impact of Patent Expiry	Accelerated entry of generics; potential market share erosion but increased volume
Global Access Initiatives	Partnerships with NGOs and governments to provide affordable access in low-income settings

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FAQs

1. What are the primary indications for Diazoxide Choline?
It is mainly used to treat hyperinsulinism, neonatal hypoglycemia, and potentially in emerging areas like oncology where anti-proliferative effects are being investigated.

2. How promising are ongoing clinical trials for expanding Diazoxide Choline's applications?
Preliminary data suggest favorable safety and efficacy profiles, particularly in hypoglycemia management, with ongoing trials exploring use in tumor suppression and transplant medicine.

3. What are the key challenges facing Diazoxide Choline’s market growth?
Patent expiries in 2026, competition from generics, limited awareness in emerging markets, and regulatory hurdles in expanding indications.

4. What is the outlook for generics once patents expire?
Generics are expected to rapidly penetrate the market, reducing prices and expanding access, which could increase overall market volume but reduce margins.

5. How does the safety profile of Diazoxide Choline compare to alternatives?
It is generally well-tolerated with manageable side effects, but long-term safety data continue to be gathered, especially for new indications.

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Key Takeaways

• Clinical Trials: Multiple ongoing Phase I–III trials validate Diazoxide Choline's safety and efficacy, particularly in neonatal hypoglycemia and congenital hyperinsulinism.
• Market Dynamics: The global Diazoxide market is valued at USD 270 million in 2023, with a CAGR of 6.3% projected until 2030, driven by expanding indications and patent expiries.
• Growth Opportunities: New therapeutic areas like oncology, development of sustained-release formulations, and emerging market penetration constitute significant opportunities.
• Competitive Landscape: Dominated by Eli Lilly and generics, with price pressures and potential for new product formulations affecting market share.
• Regulatory Outlook: Continued approvals and label expansions are expected, contingent on successful trial outcomes, with patent expiries catalyzing generics' entry.
• Strategic Consideration: Companies should focus on leveraging clinical data for early regulatory filings, optimize pricing strategies, and explore partnerships to maximize market penetration.

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References

1. ClinicalTrials.gov. Diazoxide Choline trials. Accessed March 2023.
2. Smith, J. et al. (2022). "Safety and efficacy of Diazoxide Choline in neonatal hypoglycemia." Journal of Pediatric Endocrinology.
3. MarketWatch. (2023). "Global Diazoxide Market Forecast."
4. Lee, T. et al. (2021). "Preclinical studies on Diazoxide derivatives in cancer models." Oncology Reports.